SATURDAY, NOVEMEBER 9, 2019

St. Patrick's Episcopal School, Washington DC

Black Tie

Join us

Join us for a magical night as friends, family, and community come together to achieve a cure for Annabel. Together, we can realize our dream of treating patients suffering from Annabel’s disease, and rewrite the future for thousands of other children whose lives could be transformed by gene therapy.

Annabel has a rare and severe neurological disease called Alternating Hemiplegia of Childhood (AHC).

Every few days Annabel suffers from a day of painful, debilitating episodes similar to an epileptic seizure or a stroke. Over time, Annabel’s condition will lead to permanent brain dysfunction. Many AHC patients suffer an abrupt and irreversible deterioration, which leaves them unable ever to walk or talk again. In other cases, AHC causes a failure in the autonomic nervous system that can cause sudden, inexplicable death.

Annabel’s family, in close collaboration with the two national AHC charities, has assembled a team of nationally renowned scientists and doctors to pursue a cure for AHC. The team is pursuing a cure with AAV-mediated gene therapy, and testing one mice has begun. If the tests show evidence of a rescue in the mice, as the scientists anticipate, they will achieve the pivotal “proof of concept” to open the path for clinical trial in humans.

As the scientific team makes rapid progress, we need to provide them with the resources to move forward. Annabel’s disease is so rare—literally one in a million—that funding for research has been impossible to attain. Until now.